STAT+: Pharmalittle: Sarepta Duchenne therapy misses primary endpoint; FDA holds panel for first CRISPR treatment for sickle cell
Sarepta said its gene therapy for Duchenne muscular dystrophy failed to improve muscle function compared to a placebo in a large clinical trial.
Rise and shine, everyone, another busy day is on the way. We can tell by the early hour the official mascots bounded outdoors to chase their breakfast and the increasing volume of traffic passing by the Pharmalot campus. Moreover, some may also note today is when a parade of odd-looking individuals may knock on your door. Beyond the usual cup of stimulation — our choice today is the seasonal pumpkin spice — this calls for a treat or two. Meanwhile, we have gathered another menu of tidbits for you to peruse as you attempt to manage the moment. On that note, we hope you conquer the world today. Best of luck, and do keep in touch. …
Sarepta Therapeutics said its gene therapy for Duchenne muscular dystrophy failed to improve muscle function compared to a placebo in a large clinical trial — likely a major disappointment for those who have desperately awaited the treatment for years, STAT says. The company said all patients improved and secondary measurements indicated the drug had an effect, but the results are likely to raise questions about whether the U.S. Food and Drug Administration will expand access. The FDA granted accelerated approval, but only for 4- and 5-year-olds. The study was designed to confirm a benefit in those patients while also potentially providing a basis to expand approval to older patients.
Genetic testing companies and biotechnology groups are lobbying for revisions to a new Montana law that is more stringent than other state requirements intended to protect a consumer’s genetic data privacy, including additional oversight for increasingly popular at-home kits, Bloomberg Law tells us. GSK and the coalition representing consumer genetic testing companies 23andMe and Ancestry.com are among those that argue Montana’s law imposes unworkable mandates that will impede research. The law establishes disclosure and consent requirements for how genetic data are collected, used, and shared.
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